3 terminated, 2 completed, 1 recruiting. 1 Not applicable, 1 Phase 1, 1 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Not applicable | 1 | Completed 1 |
| Phase 1 | 1 | Recruiting 1 |
| Phase 3 | 1 | Terminated 1 |
Follow Late-onset Pompe Disease: new trials by email Literature Review of Late-onset Pompe Disease trials Research Report on Late-onset Pompe Disease
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of DNL952 in Adult Participants With Late-Onset Pompe Disease DNL952 - Late-onset Pompe Disease | Phase 1 | Recruiting | Denali Therapeutics Inc. | 32 | 2026-05-12 | 2026-08-17 |
| High Dose Inspiratory Muscle Training in LOPD IMT therapy using the Pr02 mobile device - Late-Onset Pompe Disease, Lysosomal Disease | Not applicable | Completed | Duke University | 34 | 2022-07-19 | 2025-10-01 |
| LATE-ONSET POMPE DISEASE AND CEREBROVASCULAR MANIFESTATIONS Late-Onset Pompe Disease | Completed | Hospitales Universitarios Virgen del Rocío | 477 | 2020-05-01 | 2026-07-06 | |
| STRIDE Study - A Study in Subjects With LOPD Who Are Currently Being Treated With ERT Late-onset Pompe Disease | Terminated | Amicus Therapeutics | 12 | 2017-12-08 | 2025-07-08 | |
| A Study of Respiratory Muscle Strength in Patients With Late-onset Pompe Disease (LOPD) Respiratory muscle strength measurements by different techniques. - Late-onset Pompe Disease | Terminated | BioMarin Pharmaceutical | 8 | 2014-10-21 | 2023-01-09 | |
| BMN 701 Phase 3 in rhGAA Exposed Subjects With Late Onset Pompe Disease (INSPIRE Study) BMN 701 - Late-onset Pompe Disease | Phase 3 | Terminated | BioMarin Pharmaceutical | 24 | 2014-04-01 | 2018-06-14 |
Glycogen Storage Disease Type II (2)
Brain Diseases (2) · Brain Diseases, Metabolic (2) · Brain Diseases, Metabolic, Inborn (2) · Carbohydrate Metabolism, Inborn Errors (2) · Central Nervous System Diseases (2) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (2) · Genetic Diseases, Inborn (2) · Glycogen Storage Disease (2) · Lysosomal Storage Diseases (2) · Lysosomal Storage Diseases, Nervous System (2) · Metabolic Diseases (2) · Metabolism, Inborn Errors (2) · Nervous System Diseases (2) · Nutritional and Metabolic Diseases (2)
BioMarin Pharmaceutical (2) · Amicus Therapeutics (1) · Denali Therapeutics Inc. (1) · Duke University (1) · Hospitales Universitarios Virgen del Rocío (1)
United States (5) · Belgium (2) · United Kingdom (2) · Australia (1) · Canada (1) · France (1) · Germany (1) · Italy (1) · Netherlands (1) · Portugal (1) · Spain (1)
2014 (1), 2025 (1)